Extended indication Patients With Limb-girdle muscular dystrophy type 2E (LGMD2E) (Beta-sarcoglycan Deficiency) aged 4 t
Therapeutic value No estimate possible yet
Registration phase Clinical trials

Product

Active substance Bidridistrogene xeboparvovec
Domain Neurological disorders
Reason of inclusion New medicine (specialité)
Main indication Muscular diseases
Extended indication Patients With Limb-girdle muscular dystrophy type 2E (LGMD2E) (Beta-sarcoglycan Deficiency) aged 4 to 15.
Manufacturer Sarepta
Mechanism of action Gene therapy
Route of administration Intravenous
Therapeutical formulation Intravenous drip
Budgetting framework Intermural (MSZ)

Registration

Registration route Centralised (EMA)
ATMP Yes
Submission date 2021
Expected Registration 2022
Orphan drug Yes
Registration phase Clinical trials

Therapeutic value

Current treatment options Nog geen
Therapeutic value No estimate possible yet
Substantiation De eerste resultaten uit de fase 2 studie bij kinderen (n=6) beschrijft na 2 jaar een toename van rond de 50% eiwitexpressie in de spieren en een verbetering in timed test en NSAD score. Verdere resultaten dienen afgewacht te worden om te zien of er een echte meerwaarde is voor de patiënt.
Duration of treatment one-off
References NCT03652259
Additional comments Patients with diagnosis of LGMD2E will receive SRP-9.003 administered through a single systemic injection.

Expected patient volume per year

Patient volume

1 - 9

Market share is generally not included unless otherwise stated.

References Opha.net
Additional comments De prevalentie van deze deficiëntie is 1-9 op de 1.000.000. Enkel kinderen van 4 tot 15 komen in aanmerking. Dit zou betekenen dat er mogelijk ook 1 tot 9 patiënten in Nederland in aanmerking zullen komen aangezien er 1 miljoen inwoners in Nederland zijn binnen deze leeftijdsgroep.

Expected cost per patient per year

There is currently nothing known about the expected cost.

Potential total cost per year

There is currently nothing known about the possible total cost.

Off label use

There is currently nothing known about off label use.

Indication extension

Indication extension No
References Adis Insight

Other information

There is currently no futher information available.